ARINA-1 is a nebulized therapy to treat chronic inflammatory lung diseases. It is currently being developed for use in patients with primary ciliary dyskinesia (PCD) and alpha-1 anti-trypsin deficiency (A1AD) as a twice daily, chronic therapy to improve mucus clearance and quality of life. Renovion is plans to initiate a proof-of-concept studies in both indications in 2020.
Neither PCD nor A1AD have any FDA-approved therapies for pulmonary complications; all therapies used are done so off-label. Given its strong track record of safety, as well as its unique mechanism, ARINA-1 may significantly improve the lives of patients with these rare diseases.
INNATE IMMUNE MODULATION
ARINA-1 is a first-in-class therapy that addresses deficiencies in the innate immune system, the first line of defense in the lungs. In PCD and A1AD, a combination of immune defects (e.g., ciliary defects, enzyme deficiencies) and chronic environmental exposure (e.g., bacteria, viruses) result in the breakdown of the first lines of defense that prevent inflammation and infection in the airway.
ARINA-1 restores critical components to restore the innate immune system, including restoring impaired mucus clearance and directly decreasing pathological inflammation caused by neutrophil signalling and bacterial infiltration into the airway. By restoring the innate immune defenses, ARINA-1 maintains a barrier from not only inflammation within the lungs, but also environmental stresses that damage the lungs and result in clinical decline.